Don’t Miss CureDuchenne at the Gene Therapy for Muscular Disorders Event

Gene Therapy for Muscular Disorders

With recent clinical updates from Pfizer, Sarepta and Audenteson their programs in muscular disorders, the timely Gene Therapy for Muscular Disorders arrives as the only meeting dedicated to overcoming the translational and clinical challenges presented in this field.

Over 130 industry attendees are now confirmed to attend online on May 25-27, 2021.

25+ Expert Speakers:

The meeting boasts an impressive speaker faculty of industry-leaders, including:

  • Debra Miller, Founder & Chief Executive Officer, CureDuchenne 
  • Louise Rodino-Klapac, Ph.D., Executive Vice President & Chief Scientific Officer, Sarepta Therapeutics
  • Kevin Whittlesey, Vice President, Translational Medicine, 4D Molecular Therapeutics
  • Kristy Brown, Director, R&D, Solid Bio
  • Petra Kaufmann, Senior Vice President, Clinical Development/Analytics & Translational Medicine, Novartis Gene Therapies
  • Serge Braun, Chief Scientific Officer, Neuromuscular, Genethon
  • Valeria Ricotti, Co-founder, Executive Vice President & Chief Medical Officer, DiNAQOR

They’ll be joining the likes of PTC Therapeutics, Pfizer, AskBio, Audentes Therapeutics, Vertex Cell & Gene Therapies and many more pioneers of this space.

Click here to view the full list of speakers.

Unmissable Insights:

A snapshot of the topics that will be covered:

  • Optimizing gene therapy delivery with next generation capsids & muscle-specific promoters
  • Clinically meaningful endpoints & outcome measures for muscular disorders
  • Quality & quantity: addressing manufacturing challenges for large muscular disorder doses
  • Lessons learned: mitigating toxicity & immunogenicity challenges
  • Harnessing the value of preclinical model challenges to effectively determine dose
  • Navigating clinical challenges for muscular disorders

Click here to view the agenda.

The agenda also includes built-in Q&A sessions where attendees can gain additional insights directly from the speakers, just as they would at a physical conference.

Add to this the dedicated networking time with other attendees, plus a live chat function on the digital platform and the opportunities for engaging discussions are endless.

Deep Dive Discussions:

Attendees are also invited to participate in up to three pre-conference workshops:

  • Workshop A: Gene Therapy Immunogenicity: The Biology & Strategies
  • Workshop B: Exploring Advances in Next Generation Capsids & Promoters
  • Workshop C: Discovering the Latest Biology Research for Muscular Disorders

These workshops offer an exclusive opportunity to deep dive into some of these most pressing challenges in the field with like-minded professionals. They’ll be led by experts from the likes of University of Florida, University of Washington & The Ohio State University.

Click here to find out more about the meeting workshops.

Only Two Weeks to Go:

Response to the meeting has been unprecedented, with over 130 attendees from 70+ organizations already confirmed to attend.

The meeting offers group discounts of up to 20% so that delegates can attend as team.

Register here to join this meeting online on May 25-27, 2021.

Find Out More:

View the full event guide here for more details about the speakers, agenda, digital platform and pricing.

Alternatively, contact the team using the details below.


General Enquiries & Registration Assistance
Jack Gamblin
Delegate Acquisition Manager
Hanson Wade
(+1) 617 455 4188

Partnership Opportunities
Nicholas Ramovic
Partnerships Director
Hanson Wade
(+1) 617 455 4188

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